WASHINGTON (dpa-AFX) - BioMarin Pharmaceutical Inc. (BMRN) announced positive new data from studies of VOXZOGO in children with achondroplasia and in ongoing clinical trials investigating other skeletal conditions, as well as PALYNZIQ in adults with phenylketonuria. The company said the latest findings further validate the long-term benefit and established safety profiles of both medicines.
The company said new data from a study in Japan showed strong treatment adherence with VOXZOGO in children with achondroplasia under the age of 3, with no reported treatment-related adverse events nor any dose interruptions among 63 children followed for up to 23.7 months. These real-world findings validate VOXZOGO's established safety profile.
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